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How Emerging Gene Therapy Platforms Offer Hope for Leigh Syndrome PatientsLeigh Syndrome, also known as Leigh disease, is a rare, progressive, and debilitating neurodegenerative disorder primarily affecting infants and young children. Characterized by defects in mitochondrial energy production, the syndrome leads to severe neurological dysfunction, muscle weakness, developmental delays, and respiratory complications. Due to its genetic and metabolic complexity,...0 Commenti 0 condivisioni 1K Views 0 Anteprima1